UK collaboration will revolutionise rare disease treatment

rare disease treatment
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The Medicines Manufacturing Innovation Centre (MMIC) has developed a scalable, sustainable and more cost-effective way to manufacture oligonucleotides and treat rare diseases

The Medicines Manufacturing Innovation Centre (MMIC) was established by a collaboration between, UKRI, CPI, the University of Strathclyde, Scottish Enterprise and industry partners, AstraZeneca and GSK.

It has today launched a scalable, sustainable and more cost-effective way to make oligonucleotides.

Oligonucleotides are short strands of synthetic DNA or RNA that can be used to tackle both rare and more common diseases, like some types of heart disease, by interfering with how genes are expressed.

Known as the ‘Grand Challenge 3’ project, partners will employ their expertise across scale-up, analytics and process development with the goal of transforming the oligonucleotide supply chain.


Inclisiran, a small interfering RNA used in the treatment of atherosclerotic cardiovascular disease (ASCVD), will be the first medicine produced at scale.

UK Research and Innovation (UKRI) is investing £1 million into the collaboration alongside AstraZeneca, Exactmer and Novartis.

Nadhim Zahawi, Minister for Life Sciences said:

“The UK’s life sciences sector is thriving, and we’ve seen this first-hand over the past year as we’ve tackled coronavirus – from identifying crucial medicines and treatments to developing potentially lifesaving vaccines.

“As we build back better, this pioneering collaboration between industry and government will help to scale up the UK manufacture of this new class of medicines so that we can roll out crucial therapeutics to treat people with chronic diseases and help save lives.”

Life science’s champion, Professor Sir John Bell said:

“The technical challenges that this collaboration is setting out to address will, if met, be transformative. The ability to produce next-generation oligonucleotide based-medicines at scale creates enormous potential to treat a wide range of common diseases, as well as cardiovascular disease where Inclisiran clearly has a role.

“I am delighted to see this historic collaboration of industry and government launch here in the UK.”

Sarah Goulding, joint medicines manufacturing challenge director at UKRI, said:

“Oligonucleotides, are short strands of modified RNA or DNA that have wide ranging applications in advanced medicines, from genetic testing to life-saving treatments. To date, their therapeutic use has been limited to diseases impacting small numbers of patients.

“Recent developments, however, have now shown them to be effective in treating chronic illnesses that impact large numbers of people.

“Currently they’re expensive and time-consuming to produce, so we’re funding this project because it will develop technologies that enable oligonucleotides to be developed at an industrial scale, at speed and at lower cost, allowing this important development to help tackle today’s health challenges and those of the future.”


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